Recruitment completed at the Paris Institute of Myology for the EJP-DT4RD European Study

April 23 2024, 10:00 CEST

Following our last press release in 2023, the Digital Tools for Rare Disease project (DT4RD), a product of the Rare Disease Research call placed by the European Joint Programme on Rare Diseases in partnership with Fondation Maladies Rares sponsored by Chiesi and CSL Behring, reached a major milestone: The Institute of Myology announced proudly that their 20th and last patient was recruited into the study on the 29th March 2024!

We are delighted to have reached our recruitment objective. This is a major milestone. Patients have worked hard with our team of specialists. The next phase will consist of extracting and analysing as much data as possible to deeply explore the relationships between hospital and home measurements

Dr Jean-Yves Hogrel, Director of the Laboratory of Neuromuscular Physiology and Evaluation at the Institute of Myology

The main objective of the DT4RD project is to develop and validate a way to acquire, transfer, store and process heterogeneous data related to movement measured at home and anchored to assessments performed at the hospital.

The study involves the use of state-of-the-art wearable sensors integrated with Aparito’s Atom5TM eCOA platform to measure physical activity and movement at home. The platform is also used to measure a patient’s upper and lower limb function via video assessments and psychosocial parameters via its ePRO capabilities.

Patients enrolled on the study range from 24 to 60 years, including 11 men and nine women, living with neuromuscular diseases, either Becker Muscular Dystrophy (one patient), DM1 (five), FHSD (five), Congenital RYR1-associated myopathy (one), Mitochondrial cytopathies (POLG disease) (one) and LGMD (seven) – all have contributed to significant insight with more than 120 video tasks already captured, 1750 days worth of wearable and sensor data, and 100 data points across Patient Reported Outcomes.

We acknowledge the significant efforts from the Institute of Myology and the patients who agreed to join this study. Thank you! This is a significant milestone towards anchoring home vs hospital measurements in the field of neuromuscular diseases.

Dr Elin Haf Davies, CEO of Aparito

This European study involves renowned clinical experts from the Institute of Myology (Paris, France), and the John Walton Muscular Dystrophy Research Centre (Newcastle, UK). 

By digitising clinically meaningful procedures, Aparito leads this project with European clinical partners as well as renowned technology and industry partners to provide real-time visibility of data collection and integrity, reducing patient and site burden, enhancing the patient experience, and improving patient retention. This EJP-DT4RD study is supported by the Atom5TM eCOA platform’s innovative technology in the patient’s homes using a study-specific smartphone app, video assessments, and wearable technology to deliver meaningful data through eCOAs and the development of digital biomarkers.

This EJP-DT4RD study is supported by the Atom5TM platform’s innovative technology in the patient’s homes using disease-specific smartphone apps, video assessments, and wearable technology to deliver meaningful data through eCOAs and the development of digital biomarkers.


About Aparito
Aparito’s mission is to digitize clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments. Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to deliver digital clinical trials using novel eCOA to generate continuous real-world data. Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies.

Aparito logo

About the Institute of Myology

The Institute of Myology, located in Paris, coordinates medical management, research, and education related to muscle diseases and injuries. It is an international reference centre that participates in numerous trials and clinical studies, with a focus on neuromuscular diseases, high-performance sports, and ageing. The Institute of Myology Association facilitates the coordination of site activities in partnership with five public guardianship organisations, and the institute includes a complete Centre of Research in Myology and several scientific poles as well as clinical activities.

Find them on https://www.institut-myologie.org/en/

Institut de Myologie logo

About the EJP-DT4RD project

The European Joint Programme Digital Tools for Rare Disease (EJP-DT4RD) is a unique project involving Academics (Institute of Myology, John Walton Muscular Dystrophy Research Centre), SMEs (Aparito, Yumen Bionics), large pharmaceutical companies (Chiesi, CSL Behring), and a patient organisation (MSUK) working towards the same objective for and with patient involvement. It widens the horizon of tremendous possibilities for the future of research in rare diseases and warrants hope for future clinical trials using Real World data.

Find out more at https://www.ejprarediseases.org/rare-diseases-research-rdr-challenge-1-digital-tools-for-rare-disease-dt4rd/


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How to Meet Regulatory & Compliance Requirements for Capturing eCOAs in Clinical Trials

How to Meet Regulatory & Compliance Requirements for Capturing eCOAs in Clinical Trials provides in-depth insights into adapting to the evolving landscape of clinical trials technology.

We highlight the integration of Electronic Data Capture (EDC) platforms, Machine Learning, and AI in clinical management to ensure audit readiness and compliance with the latest FDA and EMA guidelines. Explore the transition from traditional Computer System Validation (CSV) to a more dynamic Computer System Assurance (CSA), focusing on risk-based approaches and minimizing extensive documentation.

In this white paper you will learn:

  1. Transitioning from CSV to CSA. Understand the shift from traditional CSV to the more dynamic CSA. Learn about the FDA’s latest guidelines which emphasize risk-based approaches, reduce the burden of documentation, and ensure the security, availability, and reliability of clinical trial systems.
  2. Adapting to New Regulatory Requirements. Discover how recent regulatory changes impact the management and execution of clinical trials, specifically the integration of Machine Learning and Artificial Intelligence in clinical trial management, and how these technologies meet FDA and EMA standards for data integrity and reliability.
  3. Implementing a Robust eQMS with AMP™. Explore how the Aparito Management Platform (AMP™) streamlines your operations, integrates risk management and ensures data integrity across every aspect of your study, keeping you audit-ready and compliant in a complex regulatory environment.

Download the white paper today to ensure your team is equipped with the knowledge to implement effective and compliant eCOA strategies in your clinical trials. Transform your processes, meet regulatory demands, and stay ahead in the fast-evolving world of clinical research.

Download the whitepaper here!

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On-demand webinar: Why vCOAs are the Greatest Value Unlock in Clinical Trials

Our webinar on the transformative potential of Video Clinical Outcome Assessments (vCOA) in real-world studies is now available for on-demand viewing!

This webinar is a must-watch for clinicians, researchers, and healthcare professionals seeking to leverage the latest advancements in digital health technology to improve patient outcomes and deepen their understanding of disease progression through innovative assessment tools.

🔍 Discover the Future of Patient Data Capture with vCOA

The webinar offers an in-depth exploration of how vCOA transforms the way patient data is captured, ensuring consistency across remote and in-person assessments. We spotlight the development and validation of the video Timed Up-and-Go (vTUG) assessment within our Atom5™ eCOA platform, a collaboration with DZNE, highlighting its pivotal role in enhancing patient care and research methodologies.

💡 What You Will Learn

1. Validation of Video-Based Outcome Assessments 

Gain insights into our rigorous process for validating the usability and feasibility of video-based assessments, ensuring they meet high standards of accuracy and reliability.

2. Real-World Deployment of vTUG for Parkinson’s Disease

Discover how integrating vTUG into everyday practice is transforming the follow-up of patients with Parkinson’s Disease, enabling continuous, real-world monitoring that captures the nuances of disease progression.

3. Automated Video Analysis for Enhanced Clinical Insights

Learn about the cutting-edge automated analysis of video recordings, which provides more reliable and objective measurements. This technology assists clinicians by introducing new metrics for evaluation and offers unprecedented insight into the progression of diseases.


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Measuring What Matters with eCOAs: A New Era of Patient-Led Innovation

In the evolving landscape of healthcare and clinical research, the traditional methodologies of assessing patient outcomes are undergoing a significant transformation thanks to the advent of electronic Clinical Outcome Assessments (eCOAs), marking a pivotal shift from conventional paper-based methods, offering myriad benefits that enhance the accuracy, efficiency, and inclusivity of clinical trials.

eCOAs promise enhanced data quality through direct source capture, real-time access for faster decision-making, and improved patient engagement through user-friendly interfaces. These advancements are not just about improving methods; they reflect a deeper commitment to patient-centred research and a push towards more rigorous, evidence-based practices in drug development.

In this white paper you will learn:

  1. How eCOAs boost data collection by enhancing data quality, patient participation, and efficiency in clinical research.
  2. The role of eCOAs in enabling diverse participants to join clinical research, breaking down barriers for those with limited access to traditional study sites.
  3. An overview of the expanded categories of COAs, including the latest FDA-guided developments, and how digital technologies are reshaping the way health outcomes are measured and analyzed

With examples like the SARAhome for ataxia and DMDhome for Duchenne Muscular Dystrophy, the paper illustrates the potential of eCOAs to capture nuanced changes in patient conditions and treatment impacts, offering a richer, more detailed view of patient experiences.

Moreover, the development of novel eCOAs and the digitisation of existing ones underscore the flexibility and adaptability of electronic assessments to meet the evolving needs of clinical research.

Download the full white paper to explore the transformative potential of eCOAs and discover how they are reshaping clinical trials.

Download the whitepaper here!

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Celebrating International Women’s Day

Acknowledging the contributions of women to the fields of Science, Technology, Engineering and Mathematics (STEM) is a crucial part of the Aparito story.

Aparito was founded by Dr Elin Haf Davies, a visionary nurse turned PhD doctor who wanted to change the patient experience of clinical trials and was frustrated by existing solutions.

But the tech industry isn’t renowned for its diversity.

Women make up just 4% of UK tech startup founders

11 out of 251 of the fastest growing tech start-ups in the UK were founded by women

Elin Haf went on to build a team that champions inclusivity and diversity. And the results speak for themselves.

Over half of the Aparito team are women

That’s nearly three times the industry average for tech businesses!
The Aparito team

Graciëlle Schutjens, our Scientific Business Director and Dr Rashmi Narayana, our Chief Scientific Officer, hold positions on the Leadership team.

80% of the Aparito Data Science team are women

Our Data Science team, spearheaded by Dr Clare Matthews and Sandra Komarzynski, continues to grow as they develop new digital endpoints.

There is always more to be done but we are proud of our commitment to furthering equal representation of women in STEM, and not just because greater female representation at senior levels within businesses makes those businesses more profitable and more socially responsible, but because it’s the right thing to do to create a more equitable society.

Five women who paved the way in STEM

Aparito stands on the shoulders of giants when it comes to innovating in STEM and so we celebrate five women whose contributions to the world made our work possible.

1. Elizabeth Garrett Anderson

The first English female doctor; Elizabeth Garrett Anderson (1836 – 1917) was an astounding and resilient woman whose plight and determination enabled other women to also achieve greatness.

Having been inspired by successful women such as Elizabeth Blackwell, the first female doctor in the US, Elizabeth Garrett Anderson opted to contradict the submissive life she was expected to lead and become a doctor. Denied entry to any medical school, she was forced to study nursing alongside male peers whose objections led to her dismissal.

After Elizabeth qualified as a doctor through the Society of Apothecaries, they immediately implemented a ban on female entrants. The sexism and adversity Elizabeth faced only fuelled her strength and resolve.

Having taught herself French in order to study at the University of Paris, Elizabeth finally earned her medical degree. However, this was still not enough to allow her onto the British Medical Register, so she established the New Hospital for Women, which was to become the London School of Medicine for Women.

Her vocal campaign efforts eventually paid off and in 1876 female entry into the profession of medicine was legalised.

Even once she had retired from medicine, Dr Anderson was still grinding down the patriarchy, becoming the first female mayor in England. She was influential in the suffragette movement and inspired her daughter, alongside many other intrepid women, to follow in her esteemed footsteps and strive toward gender equality.

2. Betsi Cadwaladr

Betsi Cadwaladr (1789 – 1860) was one of 16 children from Bala in North Wales. When Betsi grew up she became a traveller of note. She travelled around the world extensively in an age when most working-class men hardly ever moved outside the area they were born. Betsi was not officially a nurse but her various jobs involved her in nursing duties and she was convinced of the need for cleanliness as an aid to recovery from disease and illness.

In 1854, the Crimean War broke out and a total lack of care for the wounded and dying soldiers became evident. Betsi applied to join Florence Nightingale’s group of nurses to go out to help, when she arrived she demanded to be sent to the front, Nightingale thought that Betsi was argumentative and wanted nothing more to do with her.
Betsi lived and worked just behind the front line where she cared for wounded and injured soldiers. She nursed for up to 20 hours a day and usually slept on the floor with seven other nurses by which time she was over 70 years of age.

When Nightingale visited the battlefront and saw what amazing work Betsi had done, she changed her mind about a woman whom she had only previously considered as being irritating. She begged Betsi to stay on, but Betsi knew she had reached her limit by then and returned home.

Her heroic efforts in the field of healthcare were not acknowledged during her lifetime, however, she did gain some degree of recognition in 2009 when the Betsi Cadwaladr Health Board was created to include the six local health boards along the North Wales coast.

3. Ada Lovelace

Ada Lovelace (1815-1852) was unquestionably one of the most important women in science history. She was born in 1815, the daughter to Lord Byron and Lady Byron. Lady Byron believed that if she could tame Ada’s imagination, this would prevent Ada from going down the line of imaginative self-indulgence that Byron himself had.

She set out to use mathematics as the method of taming Ada’s imagination, figuring that if she could arrange for Ada to be educated in mathematics. When Ada was only 17 years old, and on the evening of 5 June 1833, she met a man who would become arguably her most important friend.

His name was Charles Babbage. He was 24 years older than her, and she quickly became fascinated, after talking to him, with his plans for building a calculating machine called a ‘Difference Engine’. The purpose of this, although Babbage never managed to complete it, was to calculate mathematical tables automatically without error.

Babbage eventually abandoned the Difference Engine in favour of the Analytical Engine: the world’s first digital computer, with a store, a processor, a memory, a sub-routing function and all the other essential features of a modern digital computer.

It is Lovelace’s work on the Analytical Engine that has led to her being known as the first computer programmer.

4. Katherine Johnson

Katherine Johnson (1918 – 2020) was a mathematician who worked on NASA’s early space missions and was portrayed by Taraji P Henson in the film ‘Hidden Figures‘.

She was one of the “computers” who solved equations by hand during NASA’s early years and those of its precursor organisation, the National Advisory Committee for Aeronautics. Johnson and other black women initially worked in a racially segregated computing unit in Hampton, Virginia, that was not officially dissolved until NACA became NASA in 1958. Signs had dictated which toilets the women could use.

She focused on planes and other research at first. But her work at NASA’s Langley Research Centre eventually shifted to Project Mercury, the nation’s first human space programme.

She and her co-workers had been relatively unsung heroes of America’s ‘Space Race But in 2015, President Barack Obama awarded Johnson (then 97 years old) the Presidential Medal of Freedom, the nation’s highest civilian honour.

5. Barbara McClintock

We live in an age where we have mapped the human genome and developed tools such as CRISPR, but all of this was possible thanks to the dedication and lifelong study into genetics by Barbara McClintock (1902 –1992) She spent her entire career analysing maize, and in the 1930s developed a staining technique that allowed her to identify, examine and describe its individual chromosomes.

Armed with her research, she was able to determine the existence of jumping genes, which are sequences of DNA that move between the genome. Jumping genes were considered junk DNA by much of the scientific community at the time. Still, McClintock pressed on and suggested they might in fact determine which of the genes in cells are switched on – vital in creating differences between cell types.

It was not until 1983, when she was awarded The Nobel Prize in Physiology or Medicine, that the scientific community began to recognise not only just how important these jumping genes are, but how much of the genome they make up – some estimates suggest they make up 40 per cent of the human genome.

McClintock also was the first to suggest the idea of epigenetics, where genes alter their activity in response to external factors, some 40 years before it was formally studied.

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Recommendations to address respondent burden associated with PROs

Patient-reported outcomes (PROs) are crucial in healthcare research, offering insights into interventions from the patient’s perspective. This study focuses on mitigating respondent burden in #PROs to enhance data quality and decision-making.

After a thorough literature review and a Delphi survey involving multi-stakeholders, the authors compiled 19 recommendations to address this issue including “where possible, consider the use of ePROs, which may help reduce respondent burden, but must be balanced with the needs and preferences of the target population” and “explore the functionality of ePROs with diverse representatives from the target population where possible”.

Abstract

Patient-reported outcomes (PROs) are increasingly used in healthcare research to provide evidence of the benefits and risks of interventions from the patient’s perspective and to inform regulatory decisions and health policy. PROs in clinical practice can facilitate symptom monitoring, tailor care to individual needs, aid clinical decision-making, and inform value-based healthcare initiatives. Despite their benefits, there are concerns that the potential burden on respondents may reduce their willingness to complete PROs, with a potential impact on the completeness and quality of the data for decision-making. We therefore conducted an initial literature review to generate a list of candidate recommendations aimed at reducing respondent burden.

A two-stage Delphi survey by an international multi-stakeholder group followed this. A consensus meeting was held to finalize the recommendations. The final consensus statement includes 19 recommendations to address PRO respondent burden in healthcare research and clinical practice. If implemented, these recommendations may reduce PRO respondent burden.


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Launch of decentralised study to validate new endpoint for Duchenne muscular dystrophy

Wrexham, UK — February 12, 2024 09:00 GMT

DMDhome, an innovative platform designed to revolutionise the assessment of Duchenne muscular dystrophy (DMD), is now part of a groundbreaking decentralised study to validate its efficacy.

Led by Prof. Laurent Servais and Charlotte Lillien, MPT, of the Specialised Translational Research Oxford Neuromuscular Group at the University of Oxford, this pioneering study represents a significant step forward in addressing the urgent need for reliable and accessible assessments in DMD, a devastating genetic muscle-wasting disease predominantly affecting young boys.

DMDhome offers a promising solution to the challenges posed by DMD assessment. By leveraging video capture and computer vision analysis, the platform enables remote monitoring of upper and lower limb function with a focus on tasks relevant to the transfer phase of the disease. This approach not only enhances the accuracy and accessibility of assessments but also provides valuable insights into disease progression and treatment efficacy.

The primary objective of the study is to monitor changes in upper and lower limb assessments over 24 months, compared with conventional assessments, with secondary objectives focusing on validating new DMDhome electronic clinical outcome assessments (eCOAs). These eCOAs will be evaluated for their test-retest reliability, sensitivity to change, and minimally clinically important differences, providing valuable insights into disease progression and treatment response.

The decentralised nature of the study allows participants to perform assessments in the comfort of their own homes, eliminating the need for frequent clinic visits and reducing the burden on patients and caregivers. Through the use of Aparito’s DMDhome app built on the Atom5™ platform, participants can easily record and upload video tasks, complete patient-reported outcome measures (PROMs), and undergo remote screening and eligibility assessments.

The study aims to enrol a diverse cohort of participants, including 28 patients in the late ambulatory stage of DMD, 16 non-ambulant patients, and 15 age- and gender-matched healthy controls. These participants will undergo a series of upper and lower limb tasks, which will be compared against established assessments such as the North Star Ambulatory Assessment (NSAA) and the Performance of the Upper Limb (PUL2.0).

The Transfer Stage is such an important time for people with Duchenne muscular dystrophy and their families. Their life changes significantly during it. It is therefore crucial that the impact of treatments during the Transfer Stage is fully understood.

DMDhome’s electronic clinical outcome assessment in a home setting will give valuable real-world insight to help with that understanding. We are very pleased that the validation study of DMDhome has now opened to recruitment and excited to see this progress to generate important data to support the wider use of this assessment in drug development.

Dr Alessandra Gaeta, Director of Research and Development at Duchenne UK

We are extremely excited to launch the recruitment for the DMDhome study, which results from longstanding co-creation efforts between Aparito and Duchenne UK to develop innovative, objective digital outcome assessments for the Duchenne community, particularly the patients in transition.

We hope that the results of this study will support the use of DMDhome eCOAs in future clinical trials to measure motor function during the transfer stage.

Dr Elisa Ferrer Mallol, Head of Patient-led eCOAs at Aparito

The study is now live, with initial participants already enrolled. Prospective participants can join the study or request more information at DMDhome or by contacting dmdhome @ aparito.com.

Moving forward, the team behind DMDhome intends to establish its video tasks as validated eCOAs, offering valuable insights into disease progression, particularly as patients transition to the non-ambulant stage of DMD. These efforts align with broader objectives to generate reliable data for healthcare decision-making and drive advancements in DMD treatment and care.


About Aparito

Aparito’s mission is to digitise clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments.

Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to deliver digital clinical trials using novel eCOA to generate continuous real-world data.

Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies.

Aparito logo

About Duchenne UK

Duchenne UK is a charity that was set up in 2012 by Emily Reuben and Alex Johnson following both of their sons being diagnosed with DMD. They set up Duchenne UK to tackle some of the big challenges in drug development in their search to accelerate the development of treatments. In 12 years, Duchenne UK has raised more than £27 million and used this money to:   

  • Fund clinical trials of medicines that are now showing promise for DMD 
  • Set up DMD Care UK, a national care programme for patients with DMD to stop patients with DMD from dying too young because they were not getting the right care. The programme establishes best practice across all the disciplines involved in DMD care and works to ensure all medical professionals and parents know precisely what treatment children and adults with DMD need. 
  • Create a DMD medical research hub with hospital sites across the country, which has led to more trials for DMD treatments than ever before.  
  • Develop innovative technologies to support the independence of people with DMD.  

You can find out more about the work of Duchenne UK at duchenneuk.org

About MDUK Oxford Neuromuscular Centre

The MDUK Oxford Neuromuscular Centre is a partnership between Muscular Dystrophy UK and the University of Oxford. It was established in January 2019 and aims to drive forward the development of novel experimental therapies and increase national clinical trial capacity in neuromuscular diseases.

Learn more about the work of MDUK Oxford Neuromuscular Centre at onmc.ox.ac.uk

Accessibility Statement for the MDUK Oxford Neuromuscular Centre Website  (www.onmc.ox.ac.uk) — MDUK Oxford Neuromuscular Centre
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SynGAP Research Fund and Dravet Syndrome Foundation Spain join the Patient Group Accelerator Programme

January 29 2024, 14:00 GMT

Aparito is pleased to welcome SynGAP Research Fund and Dravet Syndrome Foundation Spain to the Patient Group Accelerator Programme to co-develop new digital biomarkers (BMKs) and electronic clinical outcome assessments (eCOAs) to accelerate drug development in Syngap1 and Dravet syndrome.

Aparito launched the Accelerator in 2020 to provide an initiative where a tech company and the patient community, with the support of patient advocacy groups, can collaborate as peers.

Under the Accelerator framework, Aparito and patient advocacy groups work in partnership to understand and ideally fulfil patients’ needs in terms of measuring what matters most to patients and co-develop novel digital biomarkers/eCOAs.  

We will co-develop a prototype, using Aparito’s Atom5™, to test the usability of the digital technology platform and potential future utility to capture and analyse patient-relevant digital BMKs to truly embed the patients’ voice into drug development tools that could lead to the development of medicines targeting unmet patient needs.

We are very pleased to see the consolidation of the Accelerator programme with the addition of two organisations as committed and passionate as Dravet Syndrome Foundation Spain and Syngap Research Fund. We hope to further advance the field of paediatric rare epileptic encephalopathies by co-developing new digital outcome measures to be used in future clinical drug development programmes.

Dr Elisa Ferrer Mallol, Head of Patient-led eCOAs at Aparito

We are very excited to collaborate with Aparito on developing new digital biomarkers and electronic clinical outcome assessments. Therapeutic development efforts are ongoing in various biopharma and academic labs – we expect to see trial starting in the near future, and this work is critical to finding potentially new, non-seizure based, endpoints to improve overall clinical trial readiness and accelerate drug development efforts for SYNGAP1-related intellectual disability.

Michael Graglia, Managing Director at SynGAP Research Fund

Through this project, we seek not only to facilitate drug development but also to address the unmet needs of those affected by Dravet syndrome. This initiative is instrumental in capturing the multifaceted nature of Dravet syndrome. Specifically now, as we delve into the potential of disease-modifying therapies, it becomes imperative to broaden our scope beyond merely assessing seizures, and it is necessary to have tools to evaluate these other symptoms. Together, we aim to change the landscape of clinical trials, paving the way for more patient-centric approaches and ultimately improving the lives of individuals facing Dravet syndrome.

Simona Giorgi, Scientific Director at Dravet Syndrome Foundation Spain

About Aparito

Aparito’s mission is to digitise clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments.

Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to digitise clinical trials using novel eCOA and generate continuous real-world data. 

Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies. 

Discover more about how we digitise clinical trials on LinkedIn, Twitter and at aparito.com

Aparito logo

About SynGAP Research Fund

The mission of the SynGAP Research Fund (SRF) is to improve the quality of life for SYNGAP1 patients through the research and development of treatments, therapies and support systems. 

SRF was founded in the US in 2018 as a 501(c)(3) US public charity, and families created sister organizations for SRF in the UK in 2020, in Europe (Netherlands) in 2022, and in Latin America (Colombia) in 2023. 

Completely parent-led, SRF is the largest non-government funder of SynGAP research having committed over $5 million in grants. The founders cover operational costs, ensuring donations fund science & patient-related programs. SRF’s grant program awards one or two-year grants to investigators, physician residents, and clinicians interested in studying SYNGAP1. SRF grants are intended to help researchers explore novel ideas and answer open questions related to the clinical aspects of and therapies for SRID. 

SRF is a member of FasterCures, COMBINEDbrain, Global Genes Foundation Alliance, Everylife Foundation Community Congress, Epilepsies Action Network, Personalized Medicine Coalition, Rare Epilepsy Network, and the Epilepsy Leadership Council.

For more on SRF, visit curesyngap1.org or follow @cureSYNGAP1 on LinkedIn, YouTube, Instagram, Facebook, TikTok, or X.

About Dravet Syndrome Foundation Spain

Dravet Syndrome Foundation Spain is a leading patient-driven non-profit organisation whose main objective is to promote research, raise awareness and support individuals and families with Dravet syndrome, a catastrophic form of epilepsy that begins in infancy and causes cognitive, speech, behavioural, motor and sleep disorders, among other neurodevelopmental disorders. Dravet Syndrome Foundation Spain leads, supports and funds research projects ultimately aimed at improving the quality of life of those living with Dravet syndrome.

It also provides resources and support to affected individuals and families, as well as educational materials and events for professionals and the general public. Throughout its history, Dravet Syndrome Foundation Spain has received numerous awards, including the Epilepsy Award from the Spanish Society of Neurology or the Rare Disease Awareness Award from AELMHU, the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories.

For more information about Dravet Syndrome Foundation Spain, visit dravetfoundation.eu.


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A patient-centred and multi-stakeholder co-designed observational prospective study protocol: Example of the adolescent experience of treatment for X-linked hypophosphataemia (XLH)

In “A patient-centred and multi-stakeholder co-designed observational prospective study protocol: Example of the adolescent experience of treatment for X-linked hypophosphataemia (XLH)”, a diverse team of expert physicians, researchers, technology specialists, patients, and caregivers came together to co-design a prospective observational study on XLH, a rare disease affecting adolescents.

This protocol adds to the evidence that keeping patients at the heart of research design is crucial for generating robust and meaningful data.

Abstract

The importance of patient centricity and keeping the patient at the heart of research design is now well recognised within the healthcare community. The involvement of patient, caregiver and clinician representatives in the study design process may help researchers to achieve this goal and to ensure robust and meaningful data generation.

Real-world data collection allows for a more flexible and patient-centred research approach for gaining important insights into the experience of disease and treatments, which is acutely relevant for rare diseases where knowledge about the disease is more likely to be limited.

Here, we describe a practical example of a patient-centric, multi-stakeholder approach that led to the co-design of a prospective observational study investigating the lived experience of adolescents with the rare disease, X-linked hypophosphataemia. Specifically, we describe how the knowledge and expertise of a diverse research team, which included expert physicians, research and technology specialists, patients and caregivers, were applied in order to identify the relevant research questions and to ensure the robustness of the study design and its appropriateness to the population of interest within the context of the current clinical landscape.

We also demonstrate how a structured patient engagement exercise was key to informing the selection of appropriate outcome measures, data sources, timing of data collection, and to assessing the feasibility and acceptability of the proposed data collection approach.


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The Digital 1-Minute Walk Test: A Breakthrough in Digital Endpoint Innovation

January 18 2024, 10:00 GMT

In a ground-breaking effort to enhance the patient-centricity of clinical trials, Aparito has collaborated with the University of Cambridge Royal Papworth Hospital, Pulmonary Hypertension Association UK, and Garmin Health to redefine the parameters of measurement and engagement in clinical research to develop the Digital 1-Minute Walk Test (d1MWT).

The Digital 1-Minute Walk Test

Traditionally, the 6-Minute Walk Test (6MWT) has been a cornerstone in assessing patient function and prognosis. However, Dr. Joe Newman from Royal Papworth Hospital challenged the status quo by questioning the arbitrary nature of the 6MWT duration (Butland et al. 1982). The research suggests that the 6-Minute Walk Distance (6MWD) may be shortened, offering numerous advantages for both patients and research protocols.

The Studies

The first study involved 102 patients with Pulmonary Hypertension (PH) and 91 patients with Interstitial Lung Disease (ILD) and demonstrated that the distance walked in the 1st minute (1MWD) correlates nearly perfectly with the traditional 6MWT distance.

Furthermore, 1MWD and 6MWD show equivalent correlations with quality-of-life measures and predictive abilities for 3-year mortality.

The study team then used Aparito’s Atom5™ platform to gather raw data from patients with pulmonary hypertension using Garmin wearables via Garmin Health’s SDK integration to capture outdoor 1-Minute Walk Tests (1MWTs).

This technically accurate, safe, and repeatable system strongly correlates with the gold-standard 6MWT.

Patient-Centric Digital Innovation

Digital innovation plays a pivotal role in this paradigm shift: the introduction of the digital 1MWT facilitates decentralised (remote) trial delivery and reduces patients’ travel time and costs. It allows for higher frequency of testing, captures other wearable data (such as step count), and integrates seamlessly with digital Quality of Life measures (ePROMs).

“Why bother shaving off 5 minutes?” asks Dr. Newman. “The advantages are numerous; increased comfort for patients, higher adherence, greater inclusivity for those who can’t walk for 6 minutes, significant time and cost savings for physiology departments, and enhanced practicality in outdoor settings.”

Dr Newman’s research emphasises the importance of patients’ perspectives. Through extensive collaboration and feedback, it was found that patients prefer remote digital testing and shorter walk times.

This insight has led to a new digital biomarker – the d1MWT – with both technical accuracy and high patient acceptability.

Dr. Newman poses a critical question: “Could the d1MWT provide a decentralised and patient-centred solution to the need to innovate endpoints in trials of heart/lung disease and clinical practice?”

Whilst this study is undoubtedly a promising step forward for patients with pulmonary hypertension, it has the potential to help many other patients beyond this rare lung disease.

To read the paper in full please visit The Digital 1-Minute Walk Test: A New Patient-centered Cardiorespiratory Endpoint | American Journal of Respiratory and Critical Care Medicine | Articles in Press (paywall)

About Aparito

Aparito’s mission is to digitise clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments.

Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to digitise clinical trials using novel eCOA and generate continuous real-world data. 

Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies. 

Discover more about how we digitise clinical trials on LinkedIn, Twitter and at aparito.com

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